Cancer drug may reverse genetic autism

Researchers believe a cancer drug could be used to treat a genetic form of autism.

Scientists led by Professor Riccardo Brambilla, from Cardiff University, found the experimental cancer drug may permanently reverse the genetic autism.

Professor Riccardo Brambilla, who led the trial of a cancer drug on mice with a genetic form of autism

People with a missing region on chromosome 16 lack a protein called ERK1.

This missing protein causes the abnormal activation of another protein, ERK 2. This, in turn, appears to cause autism symptoms.

Cancer drug reversed autistic traits

In their experiments with mice, the scientists used the cancer trial drug to stop ERK2 reaching the brain. They found it reversed most of the autistic traits.

Brambilla said the drugs provided “symptomatic relief” in adult mice.

The drugs also prevented genetically predisposed mice being born with autism when given to the mother during pregnancy.

Doctors could treat children ‘as early as possible’

Brambilla said doctors could treat children “as early as possible after birth”. He thought, however, it would not be feasible to treat pregnant women.

Professor Jonathan Green, who was not involved in the research, specialises in child and adolescent psychiatry at Manchester University and has a research interest in autism.

He said while the paper is “interesting”, the results apply only to mice. Most findings of this kind when tried out on humans have not held up, he said.

Potential tests may take ‘many years’

Also, any potential tests on humans are likely to take “many years”.

Further, the results apply only to a “rare genetic condition”, rather than “general autism”, said Green.

Only 1 per cent of people with autism have the genetic autism linked to the chromosome 16 defect.

The research took place in collaboration with the laboratory of Professor Gary Landreth from Indiana University School of Medicine and Dr Alessandro Gozzi from the Istituto Italiano di Tecnologia. It has been published in The Journal of Neuroscience.